Positive new data for Roche ’s Evrysdi in largest trial ever undertaken in patients with previously-treated spinal muscular atrophy (SMA)

New two-year Evrysdi data show improvement or maintenance of motor function in people with SMA, a progressive neuromuscular disease that can be fatalThe JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trialLonger-term safety data consistent with that previously seen in earlier trials and low study drop-out rateEvrysdi has proven efficacy in babies, children and adults, with more than 7,000 patients treated to date worldwideBasel, 12 October 2022- Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new two-year data from the JEWELFISH study evaluating Evrysdi ® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrolment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including nusinersen (Spinraza(R)) or onasemnogene abeparvovec (Zolgensma(R)). Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after 2-years of treatment. These data will be presented at the 27th World Muscle Society (WMS) congress, 11-15  October 2022.“The consistent safety profile and exploratory efficacy we have seen in the JEWELFISH study, the largest ever conducted in previously treated patients, reinforces Evrysdi as a meaningful treatment option across SMA populations,” said Dr. Claudia Chiriboga, Professor of Neurology and Pediatrics, Department of Neurology, Columbia University Medical Ce...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news